What Is the NORD Rare Disease Database?
The NORD (National Organization for Rare Disorders) Rare Disease Database is an inclusive and comprehensive list of rare diseases. "When you hear hoofbeats, think horses, not zebras" is a medical school phrase that means doctors should consider the most likely diagnosis first, rather than the least likely. In medicine, a zebra represents a rare disease. For this reason, the zebra awareness ribbon pin represents rare diseases and all diseases in this rare disease database list.
Personalized Cause® prints the zebra design on an enamel pin that includes a rubber backing. Zebra awareness pins are available in both a non-personalized and personalized version. We also offer a zebra, pink and blue pin in a personalized and non-personalized version. This is a further reminder of the struggle people face when seeking a rare disease diagnosis. The zebra, pink and blue pins further highlight the struggle with being undiagnosed.
Different Designated Colors for the Same Disease or Disorder
Occasionally, a rare disease has a separate, designated color that is different from the zebra image. When this occurs, please feel free to use either the zebra design or designated color. The purpose of the zebra pin is to be a universal symbol of rare disease designation. Our purpose is not to be judgmental of the color you choose. Please avoid analysis paralysis and worry that you are choosing the wrong color when you make your selection. The choice is yours.
Most importantly, your purpose is to raise awareness. This is especially true for diseases and disorders that get little recognition or may hit close to home. Ultimately, our goal is to help unknown diseases and disorders gain recognition. Again, we do this with the use of a simple, but meaningful, awareness ribbon pin.
What is a Rare Disease as Designated by NORD?
A rare disease, as designated by NORD, is a disease, disorder, illness or condition that affects fewer than 200,000 Americans. In total, there are over 10,000 rare diseases that affect more than 30 million Americans. Because of this, NORD dedicates itself to the identification, treatment, and cure of these diseases. They do this through comprehensive education, advocacy, research, and service programs.
NORD rare diseases present unique challenges for researchers and companies working toward treatments and cures. This is due, in part, to:
- Small patient populations.
- Hard to diagnose or delayed diagnosis due to lack of education and understanding among medical professionals.
- Poor understanding of the natural history of the disease and its progression without intervention.
- No prior pathways to follow, including clinical endpoints that are often unclear.
- Enrollment and retention challenges.
What is an Orphan Drug?
Lastly, let's discuss the purpose of orphan drugs. An orphan drug is a drug intended to treat a condition affecting fewer than 200,000 Americans. Or, which will not be profitable within 7 years following approval by the FDA. An orphan drug is a medication used to treat, prevent, or diagnose a rare disease or condition. In other words, the term "orphan" refers to the fact that these drugs may have been abandoned or "orphaned." This is due to a lack of funding or interest in development.
Special Circumstances for Orphan Drug Approval
However, patients with rare diseases desperately need orphan drugs. The Orphan Drug Act (ODA) passed in 1983. It is designed to encourage the development of orphan drugs by providing financial incentives and other assistance to manufacturers. As a result, the FDA may provide Orphan Drug Designation to a drug or biological product that is being investigated for rare disease use. The ODA has led to the approval of more than 600 orphan drugs.
A Comprehensive List of NORD® Rare Diseases:
NORD Rare Diseases – A THRU M
| Aarskog Syndrome |
| Abetalipoproteinemia |
| Ablepharon-Macrostomia Syndrome |
| Acanthocheilonemiasis |
| Aceruloplasminemia |
| Achalasia |
| Achard Thiers Syndrome |
| Achondrogenesis |
| Achondroplasia |
| Acid Sphingomyelinase Deficiency |
| Acoustic Neuroma |
| Acquired Aplastic Anemia |
| Acquired Hemophilia |
| Acquired Lipodystrophy |
| Acquired Neuromyotonia |
| Acrocallosal Syndrome, Schinzel Type |
| Acrodermatitis Enteropathica |
| Acrodysostosis |
| Acromegaly |
| Acromesomelic Dysplasia |
| Acromicric Dysplasia |
| ACTH Deficiency |
| Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) |
| Acute Cholecystitis |
| Acute Disseminated Encephalomyelitis |
| Acute Eosinophilic Pneumonia |
| Acute Intermittent Porphyria |
| Acute Myeloid Leukemia |
| Acute Promyelocytic Leukemia |
| Acute Respiratory Distress Syndrome |
| Adams-Oliver Syndrome |
| ADCY5-Related Dyskinesia |
| Addison’s Disease |
| Adenoid Cystic Carcinoma |
| Adenylosuccinate Lyase Deficiency |
| Adie Syndrome |
| ADNP Syndrome |
| Adult Neuronal Ceroid Lipofuscinosis |
| Adult Polyglucosan Body Disease |
| Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia |
| Adult-Onset Still’s Disease |
| AEC Syndrome |
| African Iron Overload |
| Agammaglobulinemia |
| Agenesis of Corpus Callosum |
| Agranulocytosis, Acquired |
| Ahumada-Del Castillo Syndrome |
| Aicardi Syndrome |
| Aicardi-Goutières Syndrome |
| AIDS Dysmorphic Syndrome |
| ALAD Porphyria |
| Alagille Syndrome |
| Alexander Disease |
| Alkaptonuria |
| Alopecia Areata |
| Alpers Disease |
| Alpha Thalassemia |
| Alpha Thalassemia X-linked Intellectual Disability Syndrome |
| Alpha-1 Antitrypsin Deficiency |
| Alpha-Mannosidosis |
| Alport Syndrome |
| Alström Syndrome |
| Alternating Hemiplegia of Childhood |
| Alveolar Capillary Dysplasia with Misalignment of Pulmonary Veins |
| Alveolar Soft Part Sarcoma |
| Ameloblastic Carcinoma |
| Ameloblastoma |
| Amelogenesis Imperfecta |
| Amniotic Band Syndrome |
| Amniotic Fluid Embolism |
| Amyloidosis |
| Amyotrophic Lateral Sclerosis |
| Anaplastic Astrocytoma |
| Andersen Disease (GSD IV) |
| Andersen-Tawil Syndrome |
| Anemia of Chronic Disease |
| Anemia, Hemolytic, Acquired Autoimmune |
| Anemia, Pernicious |
| Anencephaly |
| Angelman Syndrome |
| Angioimmunoblastic T-Cell Lymphoma |
| Aniridia Cerebellar Ataxia Mental Deficiency |
| Anthrax |
| Antiphospholipid Syndrome |
| Antisynthetase Syndrome |
| Antithrombin Deficiency |
| Antley-Bixler Syndrome |
| AP-4-Associated Hereditary Spastic Paraplegia (AP-4-HSP) |
| Apert Syndrome |
| Aplasia Cutis Congenita |
| Apnea, Infantile |
| Appendiceal Cancer and Tumors |
| Apraxia |
| Arachnoid Cysts |
| Arginase-1 Deficiency |
| Arginine Vasopressin Deficiency |
| Arginine: Glycine Amidinotransferase Deficiency |
| Argininosuccinic Aciduria |
| Aromatic L-Amino Acid Decarboxylase Deficiency |
| Arterial Tortuosity Syndrome |
| Arteriovenous Malformation |
| Arteritis, Takayasu |
| Arthritis, Infectious |
| Arthrogryposis Multiplex Congenita |
| Arthrogryposis Renal Dysfunction Cholestasis Syndrome |
| ASAH1-Related Disorders |
| Asherman’s Syndrome |
| Asherson’s Syndrome |
| Aspartylglycosaminuria |
| Aspergillosis |
| Asphyxiating Thoracic Dystrophy |
| Astrocytoma |
| ASXL3-Related Disorder |
| Ataxia Telangiectasia |
| Ataxia with Vitamin E Deficiency |
| ATR-16 Syndrome |
| Atransferrinemia |
| Atrial Septal Defects |
| Atrioventricular Septal Defect |
| Atypical Hemolytic Uremic Syndrome |
| Auditory Neuropathy Spectrum Disorder |
| Autoimmune Blistering Diseases |
| Autoimmune Blistering Diseases |
| Autoimmune Polyendocrine Syndrome Type II |
| Autoimmune Polyglandular Syndrome Type 1 |
| Autoinflammation with Infantile Enterocolitis |
| Autosomal Dominant Hereditary Ataxia |
| Autosomal Dominant Hyper IgE Syndrome |
| Autosomal Dominant Polycystic Kidney Disease |
| Autosomal Dominant Tubulo-Interstitial Kidney Disease |
| Autosomal Recessive Hyper IgE Syndrome |
| Autosomal Recessive Hypophosphatemic Rickets Type 2 |
| Autosomal Recessive Polycystic Kidney Disease |
| Axenfeld-Rieger Syndrome |
NORD Rare Diseases – N THRU Z
| N-Acetylglutamate Synthetase Deficiency |
| Nager Syndrome |
| Nail Patella Syndrome |
| Nance-Horan Syndrome |
| Narcolepsy |
| Necrotizing Enterocolitis |
| Necrotizing Fasciitis |
| Nelson Syndrome |
| Nemaline Myopathy |
| Neonatal Cholestasis |
| Neonatal Hemochromatosis |
| Neonatal Lupus |
| Neonatal-Onset Multisystem Inflammatory Disease |
| Nephrogenic Diabetes Insipidus |
| Nephrogenic Systemic Fibrosis |
| Neu Laxova Syndrome |
| Neuroacanthocytosis |
| Neurofibromatosis 1 |
| Neuroleptic Malignant Syndrome |
| Neuromyelitis Optica Spectrum Disorder |
| Neuropathic Ocular Pain |
| Neuropathy, Congenital Hypomyelination |
| Neurotrophic Keratitis |
| Nevoid Basal Cell Carcinoma Syndrome |
| Nevus Sebaceus Syndrome |
| NF2-Related Schwannomatosis |
| NGLY1 Deficiency |
| Niemann Pick Disease Type C |
| Nocardiosis |
| Non-24-Hour Sleep-Wake Disorder |
| Nonketotic Hyperglycinemia |
| Nontuberculous Mycobacterial Lung Disease |
| Noonan Syndrome |
| Noonan Syndrome with Multiple Lentigines |
| Norrie Disease |
| NORSE (New Onset Refractory Status Epilepticus) and FIRES (Febrile Infection-Related Epilepsy Syndrome) |
